In the 45 mg cohort, 100% of patients achieved a complete response
In the 15 mg cohort, 80% of patients achieved a complete response and 20% achieved a partial response
Safety was consistent with prior dose escalation study and PK analysis confirmed therapeutic exposures
Praxis plans to initiate an efficacy study in focal onset epilepsy in the second half of 2024
“The strength and consistency of response across both study arms, combined with a continued positive tolerability and safety profile, build on our earlier conviction that
Summary of Response | |||
Cohort | Partial Response Rate | Complete Response Rate | Total Response |
15mg | 20% (1) | 80% (4) | 100% (5) |
45mg | -- | 100% (3) | 100% (3) |
Combined | 12% (1) | 88% (7) | 100% (8) |
About the Phase 2a PPR Study
- Patient EEG signatures were assessed at defined measurement points over a 24-hour period after receiving placebo or
PRAX -628, and results were compared to baseline. - Patients must have demonstrated PPR during screening and baseline to be evaluable.
- A total of six patients were baselined in the 15 mg cohort, of whom five were evaluable. One patient in the 15 mg cohort did not present adequate PPR at baseline to be evaluated.
- Four patients were baselined in the 45 mg cohort, of whom three were evaluable. One patient in the 45 mg cohort was not evaluable due to lack of eligibility.
- Three patients from the 15mg cohort participated in the 45mg cohort after a washout period of >100 days.
- Three patients were on background anti-seizure medications (ASMs).
- Response Assessment
- Complete: Reduction to zero in the number of generalized PPR events at any assessment period vs. baseline.
- Partial: Reduction, other than to zero, in the number of generalized PPR events at any assessment period vs. baseline.
- Safety and PK samples were collected during the entire observation period.
- The Phase 2a study builds on positive results from animal studies and the Phase 1 dose escalation study in healthy volunteers.
PRAX -628 demonstrated unprecedented pre-clinical efficacy in the maximal electroshock seizure (MES) model.PRAX -628 was generally well-tolerated at all tested doses in the Phase 1 study.- Pharmacokinetic data from the Phase 1 study demonstrated dose-dependent exposure supporting once-daily dosing without titration to achieve potentially therapeutically effective drug concentration levels.
- Further analysis of patients in the Phase 1 study using quantitative EEG data showed a pharmacodynamic effect at all dose levels and was significantly different from placebo.
Praxis will host a call on
The live webcast and replay will be available through the Events & Presentations page of the Investors and Media section of the company’s website at www.praxismedicines.com.
About
About Praxis
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the anticipated timing of Praxis’ clinical trials, the development of Praxis’ product candidates and the potential therapeutic effects of Praxis’ product candidates, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.
The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended
Investor Contact: Praxis Precision Medicines investors@praxismedicines.com 857-702-9452 Media Contact:Dan Ferry Life Science Advisors Daniel@lifesciadvisors.com 617-430-7576
Source:
2024 GlobeNewswire, Inc., source